The purpose of this study is to understand DMD functional losses or abilities and their association with independence and quality of life from the perspective of individuals with DMD and/or and their caregivers.
This is a qualitative interview study in which individuals with DMD and/or their caregivers will be asked to participate in a semi-structured, approximately 60- minute interview. Interviews will focus on functional abilities and independence. Caregivers and boys with DMD will be interviewed. This study includes no treatment nor intervention; however, some participants are being treated by a drug that is approved in the U.S. and the U.K. and under investigation in other geographies.
Eligibility
Sex
MALE
Min age
10 Years
Max age
—
Healthy volunteers
No
Inclusion Criteria:
Subsample A - Patients
Eligible participants for Subsample A must:
* Have been treated with Givinostat for at least 2 years
* Be at least 10 years of age with signed consent of a parent or legal guardian
* Be currently taking givinostat
* Willing and able to participate in a video and/or audio recorded interview
Subsample A - Caregivers
Eligible caregivers for Subsample A must:
* Be a parent or legal guardian of an individual with DMD that has been taking givinostat for at least two years
* Willing and able to participate in a video and/or audio recorded interview
Subsample B - Caregivers
Eligible caregivers for Subsample B must:
* Provide care to an individual with DMD who is unable to raise their hands above their head as confirmed by the caregiver
* Reside in the United States or Canada
* Able to read, speak, and understand English
* Willing and able to participate in a video and/or audio recorded interview Have access to a stable internet connection
Exclusion Criteria:
\-
Primary outcome measure(s)
Qualitative Interview — 60 minutes Participants will participate in a semi-structured interview
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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